A Retrospective Case Series Describing the Efficacy of Omalizumab in Systemic Mastocytosis

Abstract Introduction Systemic mastocytosis (SM) represents a heterogeneous group of disorders characterized by accumulation of neoplastic mast cells (MCs) in one or more organ systems. Patients with SM present with a broad range of symptoms resulting from excessive mast cell mediator release, especially histamine, that frequently overlap with those of allergic disease. Episodes of life-threatening anaphylaxis are a recognized feature of SM. Here at St. Michael's Hospital we currently follow more than 50 SM patients, the largest patient cohort in Canada. Omalizumab is a subcutaneously administered monoclonal antibody which acts on circulating IgE, reducing binding to the high-affinity IgE receptor (FCεR1) on mast cells, thereby reducing the potential reactivity of these cells. At St. Michael's Hospital, omalizumab is used as an add-on, off-label therapy in SM patients at risk for recurrent anaphylaxis. The efficacy of omalizumab treatment for SM patients remains unclear. Typically, highly symptomatic patients who are refractory to all other medication are candidates for omalizumab therapy. Objectives Our primary objective was to describe the response to treatment by omalizumab in patients with SM in a tertiary care centre. Our secondary objective was to compare the markers of disease in SM patients between those who were non-responsive versus responsive to omalizumab. The clinical and biological markers to be studied are symptoms and tryptase levels. Methods This is an observational, retrospective study (n=6) of SM patients treated with omalizumab at St. Michael's Hospital between January, 2014 and June, 2018. Electronic medical records were reviewed for mastocytosis treatment, symptom progression and tryptase levels, if available. All patients included in the study were diagnosed with SM according to the 2016 WHO criteria by undergoing a bone marrow biopsy. A baseline was established 2-5 months pre omalizumab exposure, as well as two follow-ups, each ranging from 2-8 months post omalizumab exposure (av. 4.7 months). The Brown Anaphylaxis score was used to capture severity of anaphylaxis. Mild (1), moderate (2), and severe (3) scores were associated with cutaneous manifestations, systemic (GI, respiratory, cardiovascular) involvement and systemic (hypoxia, hypotension, neurological compromise) collapse, respectively. Results Our study consisted of 4 females and 2 males, with an average age of 49 years old [IQR 36-74]. All 6 patients were diagnosed with indolent SM, the more moderate of the six SM subtypes. In every system, except for respiratory, it appears that symptoms decreased once therapy began. From baseline to first follow-up: all three patients who were experiencing systemic symptoms, three of the six manifesting cutaneous symptoms, and two of the three with cardiovascular involvement, responded fully to treatment. At second follow-up, patient 1 presented to clinic asymptomatically. Overall, 100% of patients responded to treatment with responses ranging from 17% to 100% improvement of mastocytosis-related symptoms. The grading of anaphylaxis severity reported three of the six patients improving from scores of 3 (severe) to 1 (mild). The other three patients remained at scores of 2. Patients 1 and 2 (only patients with available tryptase levels at both baseline and follow-up) saw a decrease in tryptase level from 134 to 84.1 and 11.4 to 8.3, respectively. Conclusions Omalizumab appears to be an effective therapy for patients with SM with anaphylaxis and reduces tryptase levels. It should be readily considered in the management of this population. Next steps include following these patients prospectively to better capture the efficacy of omalizumab within this population. Disclosures No conflicts of interest to declare Disclosures No relevant conflicts of interest to declare.

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A Retrospective Case Series Describing the Efficacy of Ketotifen in Systemic Mastocytosis

Blood ◽

10.1182/blood-2018-99-120112 ◽

2018 ◽

Vol 132 (Supplement 1) ◽

pp. 5474-5474

Author(s):

Calum Anthony Slapnicar ◽

Conrad Scullion Smeenk ◽

Lisa K. Hicks ◽

Jessica Petrucci ◽

Peter Vadas ◽

...

Keyword(s):

Mast Cell ◽

Conflicts Of Interest ◽

Systemic Mastocytosis ◽

Tertiary Care ◽

Case Series ◽

Small Sample ◽

Mast Cell Activation ◽

Response To Treatment ◽

Mast Cell Stabilizer ◽

Organ Systems

Abstract Introduction Systemic Mastocytosis (SM) represents a heterogeneous group of disorders characterized by the accumulation of neoplastic mast cells (MCs) in one or more organ systems. Patients with SM present with a broad range of symptoms resulting from excessive mast cell mediator release, especially histamine that frequently overlap with those of allergic disease. Episodes of life-threatening anaphylaxis are a recognized feature of SM. Here at St. Michael's Hospital, we currently follow more than 50 SM patients, the largest patient cohort in Canada. Ketotifen is a dual action mast cell stabilizer used to treat asthma, anaphylaxis and other mast cell, allergic-type disorders. Along with cromolyn, another mast cell stabilizer, ketotifen is known to be the most effective at controlling mast cell activation. At St. Michael's Hospital, ketotifen is used as an add-on, off-label therapy in SM patients at risk for recurrent anaphylaxis. The literature has yet to confirm the efficacy of using ketotifen to treat SM. Few centres besides St. Michael's use ketotifen as a potential therapy. Objectives Our primary objective was to describe the response to treatment by ketotifen in patients with SM in a tertiary care centre. Methods This is an observational, retrospective study (n=10) of SM patients treated with ketotifen at St. Michael's Hospital from January 2015 to June 2018. Electronic medical records were reviewed for mastocytosis treatment and symptom progression. All patients included in the study were diagnosed with SM according to the 2016 WHO criteria by undergoing a bone marrow biopsy. Symptoms were assessed at baseline (2-5 months pre-ketotifen initiation) and again at follow-up (4-8 months post initiation). An independent statistician produced binomial proportion confidence intervals for each reported symptom. Results Our study consisted of 7 females and 3 males, with an average age of 49 years old [IQR 33-67]. 5 of the 10 patients were diagnosed with indolent SM, the more moderate of the six SM subtypes. Table 1 demonstrates the 5 most frequently reported symptoms at baseline. (Refer to Table 1) For nausea/vomiting, presyncope/syncope and urticaria, 100% of patients who reported these symptoms responded to treatment. For diarrhea and flushing, 86% and 67% of patients who reported these symptoms responded to treatment. Conclusions Ketotifen appears to be an effective therapy for patients with SM with anaphylaxis. It should be readily considered in the management of this population. Further investigation is warranted given our small sample size and single centre recruitment. Next steps include following these patients prospectively to better capture the efficacy of ketotifen within this population. Disclosures No conflicts of interest to declare Disclosures No relevant conflicts of interest to declare.

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Place of cytology in the diagnosis of systemic mastocytosis: A case report

GSC Advanced Research and Reviews ◽

10.30574/gscarr.2021.7.2.0096 ◽

2021 ◽

Vol 7 (2) ◽

pp. 01-05

Author(s):

W. Quiddi ◽

H. Boumaazi ◽

S. Ed-dyb ◽

H. Yahyaoui ◽

M. Aitameur ◽

...

Keyword(s):

Mast Cells ◽

Hematological Malignancies ◽

Systemic Mastocytosis ◽

Bone Marrow Involvement ◽

Acute Leukemias ◽

Activating Mutations ◽

Myeloid Neoplasms ◽

Organ Systems ◽

Cutaneous Mastocytosis

Mastocytosis is a heterogeneous group of rare diseases related to the clonal, neoplastic proliferation of morphologically and immunophenotypically abnormal mast cells, that accumulate in one or more organ systems. Their pathophysiology is dominated by activating mutations in C-Kit (Stem Cell Factor receptor). Several pathological forms have been described ranging from isolated cutaneous mastocytosis affecting mainly children, to aggressive systemic mastocytosis described mainly in adults with bone marrow involvement. According to the WHO 2016 classification of hematological malignancies, systemic mastocytosis appear as a new entity of "myeloid neoplasms and acute leukemias" that combines cytology (abnormal mast cells) with other genetic and molecular criteria. We describe through this observation the practical side of hematological cytology in the diagnostic orientation of this serious, rare and underestimated pathology.

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Middle East Respiratory Syndrome (MERS) Infection in Hematological and Oncological Patients: A Case Series from a Tertiary Care Center in Saudi Arabia

Blood ◽

10.1182/blood-2019-123253 ◽

2019 ◽

Vol 134 (Supplement_1) ◽

pp. 5871-5871 ◽

Cited By ~ 1

Author(s):

Ahmed Alaskar ◽

Mohammed Bosaeed ◽

Hina Rehan ◽

May Anne Mendoza ◽

Bader Alahmari ◽

...

Keyword(s):

Saudi Arabia ◽

Middle East ◽

Clinical Presentation ◽

Conflicts Of Interest ◽

Tertiary Care ◽

Case Series ◽

Case Fatality ◽

Middle East Respiratory Syndrome ◽

Future Research ◽

Fatality Rate

We present the largest to date of a case series of nine patients with hematological and oncological malignancies who were infected with Middle East Respiratory Syndrome Coronavirus (MERS-CoV). MERS-CoV is a novel beta-coronavirus with a high fatality rate in comorbid patients. The majority of MERS cases globally were reported from Saudi Arabia (1983 cases, including 745 related deaths with a case-fatality rate of 37.5%) according to the WHO update of February 2019. All were clinically stable before acquiring the virus. Most of the cases had an active disease as relapse or refractory with three cases being neutropenic. The clinical presentation and radiological features of the patients were variable and inconsistent (Table 1). Diagnosis was confirmed with RT-PCR assays targeting upstream of the E gene and the open-reading frame gene 1a which had to be done repeatedly and required an average of 3 (with max. of 7) samples for a test to be positive (Table 2). All the patients developed respiratory failure, were admitted to the critical care unit (ICU) and required mechanical ventilation. The length of hospital stay ranged from 15 - 48, with an average of 24 days. Unfortunately, all nine patients died within days after admission to the ICU. In addition, the time from diagnosis to death has an average of 9 days ranging from 2-24 days, respectively. In conclusion, MERS CoV infection in hematology/oncology patients has a very poor prognosis regardless of the status of the underlying disease. The clinical presentation is not distinctive and confirming the diagnosis requires numerous respiratory samples. Measures to prevent nosocomial outbreaks should include proper compliance with personal protection equipment by health-care workers when managing patients with suspected and confirmed MERS-CoV infection and prompt isolation of infected patients. Future research is required to enhance our understanding of the disease and to evaluate superior diagnostic and therapeutic options. Disclosures No relevant conflicts of interest to declare.

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Surgical outcome of isolated congenital supravalvular pulmonary stenosis: a case series

European Heart Journal - Case Reports ◽

10.1093/ehjcr/ytz012 ◽

2019 ◽

Vol 3 (2) ◽

Cited By ~ 2

Author(s):

Vikas Kumar ◽

Sachin Mahajan ◽

Vivek Jaswal ◽

Shyam Kumar Singh Thingnam

Keyword(s):

Pulmonary Stenosis ◽

Main Pulmonary Artery ◽

Tertiary Care ◽

Case Series ◽

Surgical Correction ◽

Medium Term ◽

Constriction Band ◽

Supravalvular Stenosis ◽

Mid Term Results

Abstract Background Supravalvular stenosis of main pulmonary artery is a rare anomaly characterized by the presence of constriction band just above the pulmonary valve. It is mostly acquired after intervention on the pulmonary trunk or less commonly is congenital in origin associated with complex congenital cardiac malformations and very rarely can present as an isolated native congenital supravalvular pulmonary stenosis (SPS). Case summary We present a series of four cases of isolated congenital SPS who underwent surgical correction at our tertiary care institute over 8 years. Mean age of the patients was 2.25 ± 0.96 years with all of them being males. Mean peak systolic gradient across the stenosis was 82 ± 21.48 mmHg ranging from 60 mmHg to 110 mmHg. There was no early and medium-term mortality with 100% survival at mean follow-up of 31 months (range 7–85 months). Discussion Surgical correction of congenital SPS carries excellent early and mid-term results with almost no mortality and very low risk of re-intervention for restenosis.

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Laser Therapy for Refractory Myringitis in Children

Otolaryngology ◽

10.1067/mhn.2002.127628 ◽

2002 ◽

Vol 127 (3) ◽

pp. 163-168 ◽

Cited By ~ 9

Author(s):

Frank P. Fechner ◽

Michael J. Cunningham ◽

Roland D. Eavey

Keyword(s):

Laser Treatment ◽

Tertiary Care ◽

Case Series ◽

Laser Technique ◽

Retrospective Case ◽

Tympanic Membrane Perforations ◽

Specialty Hospital ◽

Associated Symptoms ◽

Initial Results

OBJECTIVE: We present the application and initial results of a CO2 laser technique for the treatment of medically refractory chronic granular myringitis (CGM). STUDY DESIGN AND SETTING: Retrospective case series of 15 treated ears in 13 consecutive patients between the ages of 6 and 14 years (median age, 9.0 years) cared for in a tertiary care specialty hospital. RESULTS: Eleven of 15 treated ears had total resolution of CGM and associated symptoms; median follow-up time was 10 months. Three ears were improved, and 1 ear remained unchanged. Three of 5 preoperative tympanic membrane perforations healed after laser treatment; 1 patient developed a postoperative, dry perforation. Hearing was not impaired in any patient tested. CONCLUSION: Preliminary results suggest that CGM, when refractory to medical treatment, can often be treated effectively by a single laser treatment.

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Long-Term Hearing Outcomes following Total Ossicular Reconstruction with Titanium Prostheses

Otolaryngology ◽

10.1177/0194599819831284 ◽

2019 ◽

Vol 161 (1) ◽

pp. 123-129 ◽

Cited By ~ 4

Author(s):

C. Burton Wood ◽

Robert Yawn ◽

Anne Sun Lowery ◽

Brendan P. O’Connell ◽

David Haynes ◽

...

Keyword(s):

Care Center ◽

Tertiary Care ◽

Case Series ◽

Ossicular Chain ◽

Tertiary Care Center ◽

Ossicular Chain Reconstruction ◽

The Mean ◽

Hearing Outcomes

Objective(1) Characterize a large cohort of patients undergoing total ossicular chain reconstruction with titanium prosthesis. (2) Analyze long-term hearing outcomes of the same cohort.Study DesignCase series with chart review.SettingTertiary care center.Subject and MethodsThis study reviews patients who underwent total ossicular chain reconstruction (OCR) with titanium prostheses (TORPs) at a single tertiary care center from 2005 to 2015. Patient charts were reviewed for demographic data, diagnosis, and operative details. Patients were included in statistical analysis if length of follow-up was 2 years or more. Evaluation of hearing improvement was made by comparing preoperative air-bone gap (ABG) and ABG at follow-up at 2 years.ResultsIn total, 153 patients were identified who met inclusion criteria. The mean age of included patients was 40 years (range, 6-89 years). Sixty patients (39%) had a history of OCR, and 120 patients (78%) had a diagnosis of cholesteatoma at the time of OCR. Preoperatively, the mean ABG was 36 ± 12, whereas the mean ABG at 2-year follow-up improved to 26 ± 13. This was statistically significant ( P < .0001) using a Wilcoxon matched-pairs signed rank test. Twelve patients (8%) required revision OCR. Two revisions were performed due to prosthesis extrusion (<1%).ConclusionTitanium prostheses lead to significant improvement in hearing over long periods. The results are sustained as far out as 5 years following surgery. In addition, rates of revision surgery with titanium TORPs are low. Based on this series, there are no readily identifiable predictors for outcomes following total OCR.

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Laryngotracheal Reconstruction in Adults Aged 60 Years and Older

Otolaryngology ◽

10.1177/0194599818825471 ◽

2019 ◽

Vol 160 (6) ◽

pp. 1065-1070 ◽

Cited By ~ 3

Author(s):

Saad C. Rehman ◽

Deborah X. Xie ◽

James R. Bekeny ◽

Alexander Gelbard ◽

Christopher T. Wootten

Keyword(s):

Complete Blood Count ◽

Tertiary Care ◽

Case Series ◽

Tracheal Resection ◽

Distribution Width ◽

Free Survival ◽

Laryngotracheal Reconstruction ◽

Airway Reconstruction

Objective The primary aim of this study is to evaluate the safety, efficacy, and execution of major open laryngotracheal operations for patients in the advanced decades. Study Design Case series with chart review. Setting Multidisciplinary clinic at a tertiary care academic hospital. Subjects and Methods Patient characteristics, operative course, and postoperative outcomes were retrospectively recorded for all airway reconstruction operations performed between 1999 and 2016 on patients aged ≥60 years Long-term success was defined as prosthesis-free survival at last follow-up. Descriptive statistics were performed. Results Twenty-nine patients met inclusion criteria, and the median age was 71 years (interquartile range, 63-74). Tracheal resection was the most common procedure (13 patients), followed by laryngotracheal reconstruction (7 patients). Fifteen patients began their operation with a tracheostomy, 6 of whom underwent decannulation prior to leaving the operating room. Three additional patients underwent decannulation at follow-up appointments and were prosthesis-free at most recent follow-up. The mean time to decannulation among these patients was 3 months. Of the 14 patients beginning their procedure without a tracheostomy, only 2 required permanent airway prosthesis. The overall long-term rate of prosthesis-free survival was 72.4% (21 of 29 patients). Factors suggestive of long-term success include lower McCaffrey grade and lack of pulmonary disease, hypertension, or diabetes, as well as decreased red blood cell distribution width on preoperative complete blood count. Conclusion Through careful patient selection, preoperative workup, and meticulous postoperative care, airway reconstruction procedures in patients aged ≥60 years are reasonably successful. Of 29 patients, 21 (72.4%) were successfully breathing long-term without airway prosthesis.

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Treatment of Burkitt's Like Lymphoma: A Single Institution Case Series

Blood ◽

10.1182/blood.v118.21.4962.4962 ◽

2011 ◽

Vol 118 (21) ◽

pp. 4962-4962

Author(s):

Vishal Ranpura ◽

Shilpan S. Shah

Keyword(s):

Central Nervous System ◽

Bone Marrow ◽

Nervous System ◽

Peripheral Blood ◽

Chemotherapy Regimen ◽

Conflicts Of Interest ◽

Case Series ◽

B Cell Lymphoma ◽

Response To Treatment ◽

Single Institution

Abstract Abstract 4962 Background: Burkits's like (BL) lymphoma is a new pathologic entity with features partially resembling to Burkitt's lymphoma and diffuse large B cell lymphoma. The incidence of BL lymphoma is infrequent and may present with lymph node enlargement, involvement of bone marrow and peripheral blood. The natural history of BL lymphoma and its treatment remains unclear. Here we present a single institution case series of five patients diagnosed with BL lymphoma and their treatment. Methods: We searched the pathology reports of all patients diagnosed with lymphoma over the last one year. No inclusion or exclusion criteria were used. Results: We identified a total of five patients with diagnosis of BL lymphoma. All patients presented with lymphadenopathy and with no involvement of bone marrow, central nervous system or peripheral blood. Table 1 summarizes characteristics of all patients, their chemotherapy regimen and subsequent response to treatment. Conclusion: BL lymphoma is a new pathologic entity with low incidence. Treatment with DA-EPOCH and R-HyperCVAD has very good response rate. The data is limited by single institution case series and limited follow up time. Further studies are recommended to evaluate optimal chemotherapy regimen. Table 1: Characteristics, presentation, treatment and response in patients with BL lymphoma No Age Sex LDH, Presentation Treatment Response Duration of response 1 68 F 386 Parotid and abdominal lymphadenopathy DA-R-EPOCH CR 16months 2^ 44 M 536 Neck, Axillary, media, abdomen pelvis and sacral mets R-HyperCVAD PR 3months 3* 66 F 310 Inguinal and pelvic lymphadenopathy R-HyperCVAD f/b Autologus BMT, CR 14months 4** 71 M 237 Parotid mass 3 cycles of DA-EPOCH f/b 3 cycles of R-CHOP CR 9months 5^ 52 M 186 Neck Lymphadenopathy R-HyperCVAD CR 3months DOX, DOXOL, and anthracenediones in soluble fractions of human myocardial strips after sequential DOX loading/clearance and anthracenedione treatment Notes: DA-REPOCH: Dose Adjusted Rituximab-Etoposide, Vincristine, Cyclophosphamide, Doxorubicine, Prednisone, R-HyperCVAD: Rituximab, hyperfractionated Cyclophosphamide, Vincristine, Doxorubicin, Dexamethasone, CR: complete remission, BMT: bone marrow transplantation, R-CHOP: rituximab, doxorubicin, cyclophosphamide, vincristine and prednisone. CNS: central nervous system * patient underwent bone marrow transplant because of relapse at current presentation. ** patient was changed from DA EPOCH to R-CHOP as patient was not able to tolerate EPOCH ^ Epatients are halfway through their treatment cycles and are actively getting treatment Disclosures: No relevant conflicts of interest to declare.

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Treatment of Advanced Systemic Mastocytosis with PKC412: The French Compassionate Use Programme Experience and Historical Comparison

Blood ◽

10.1182/blood.v124.21.3193.3193 ◽

2014 ◽

Vol 124 (21) ◽

pp. 3193-3193 ◽

Cited By ~ 3

Author(s):

Marie-Olivia Chandesris ◽

Gandhi Damaj ◽

Danielle Canioni ◽

Chantal Brouzes ◽

Laure Cabaret ◽

...

Keyword(s):

Haemoglobin Level ◽

Conflicts Of Interest ◽

Systemic Mastocytosis ◽

Control Group ◽

Compassionate Use ◽

Tryptase Level ◽

Historical Comparison ◽

Significant Difference ◽

Median Haemoglobin Level

Abstract Background Advanced Systemic Mastocytosis (adSM) still have very poor prognosis. No standard of care is yet available. Midostaurin (PKC412) is a powerful TKI inhibit of VEGFR, FLT3 and most importantly the mutated CKITD816V and D816Y. Preliminarily results from an international phase II trial (Gotlib et al., Blood 2012) reported interesting results in this subgroup of patients. We report herein on the French experience of the compassionate use of PKC412 for the treatment of adSM and compare their outcome to matched adSM historical control patients (pts) who did not receive PKC412 (control group). Methods Over a period of 12 months (from August 2012, date of PKC412 Temporary Approval for Use in France), 22 pts received PKC412 and could be analysed. PKC412 100 mg twice daily was administered orally as continuous 28-day cycles until progression or unacceptable toxicity. Pts were evaluated according to the response criteria used in the recent phase 2 trial on midostaurin (Gotlib et al.). The overall survival (OS) of these 22 patients was compared to the OS of 42 age and WHO subcategories matched control adSM patients who were treated before PKC412 era. Results 2 female and 20 male pts with a median age of 65 years [35-84] received midostaurin. Overall, 21 pts (95%) with adSM (5 ASM; 2 MCL; 1 MCS; 12 ASM-AHNMD; 1 SSM-AHNMD,1) and 1 pt progressive SSM. Median number of C-Findings was 3 (0-4). Hepato- and/or splenomegaly were present in 20 (91%) and 21 (95%) pts. Median tryptase level was 204 ng/l (85-2000). Median haemoglobin level, platelet and PMN counts were respectively 10.1g/dl (6.8-13.9), 117G/L (22-289) and 2.8G/L (0.3-17). Twenty (91%) pts had cKIT D816V mutation, 2 pts had cKITWT. ASXL1 and TET2 were mutated in 3/7 and 2/7 tested, respectively. Steroids, 2-chlorodeoxyadenosine and Interferon were administered in 9, 4 and 2 pts prior to midostaurin. After a median exposure time of 6.9 [1.6-27.5] months, the overall response rate (ORR) was 77.2% including, major responses (n=13; 59%) (incomplete major in 8 pts, pure clinical in 5); partial response (n=5, 23%) (good partial in 3, minor partial in 2). Three patients were rapidly progressive (<2 months) and considered refractory to PKC412 (1 MCL, 1 MCS, 1 ASM-AHNMD). Death occurred in 4 pts (refractoriness 2 pts, AHNMD progression 2 pts). After a median follow-up of 7.4 months from PKC412 start, median OS was 24.4 months [12.6-34.2]. Patients with ASM seemed to better benefit from PKC412, however, no significant difference was found between the ASM and SM-AHNMD, probably due to the low patient’s number. The control group consisted in 42 pts with a median age of 65.5 years [23-84]. There were 17 ASM-AHNMD, 21 ISM-AHNMD, 2 SSM-AHNMD, 1 CM-AHNMD and 1 MCL patients. Median tryptase level was 107ng/l [19-501]. Median haemoglobin level, platelet and PMN counts were respectively 12.1g/dl [8-15], 200G/L [10-1036] and 4.6G/L [0.5-24.3]. Thirty-three (79%) pts had cKIT D816V mutation, 7 pts were cKITWT. ASXL1 and TET2 were mutated in 6/29 (21%) and 7/29 (24%) of pts tested, respectively. Steroids, 2-CdA, interferon, imatinib, thalidomide have been used in 19, 21, 6, 4, and 6 pts respectively. Survival distributions were estimated using the adjusted Kaplan-Meier method from the date of diagnosis to the last follow-up for the PKC412 treated and the control groups. Median survival time for PKC412 treated patients was significantly longer than control patients (p=0.04). Conclusion PKC412 is active in advanced SM. Despite the absence of complete remission, PKC412 gives survival advantage for patients with adSM over pts who did not receive the drug. Disclosures No relevant conflicts of interest to declare.

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Carcinoids of the ampulla: Long-term follow-up after endoscopic resection.

Journal of Clinical Oncology ◽

10.1200/jco.2019.37.4_suppl.430 ◽

2019 ◽

Vol 37 (4_suppl) ◽

pp. 430-430

Author(s):

George Nyasha Baison ◽

Nadav Sahar ◽

Morgan M Bonds ◽

Janelle F Rekman ◽

Flavio G. Rocha ◽

...

Keyword(s):

Endoscopic Resection ◽

Care Center ◽

Benign Lesion ◽

Tertiary Care ◽

Case Series ◽

World Health ◽

Long Term Follow Up ◽

Well Differentiated

430 Background: Neuroendocrine tumors (NET) or carcinoids of the ampulla are exceedingly rare in comparison to duodenal NET. Surgical management is widely accepted as the treatment of choice, but for patients that refuse surgery or are poor operative candidates, endoscopic resection may be option. We present a consecutive case series at a tertiary care center describing our experience with endoscopic resection of ampullary NET. Methods: This is a restrospective review with a long-term follow-up of patients with ampullary NET that were endoscopically resected. Outcomes were analyzed based on the histopathologic classification system proposed by the World Health Organization in 2000. Results: Twelve patients (9 male, 3 female), ranging in age from 41 to 86 (mean 59) underwent endoscopic ampullectomy for ampullary NET, with a mean follow-up time of 5 years. Patients had refused surgery or were poor surgical candidates. All, but one incidentally found case, were symptomatic on presentation, with gastrointestinal bleeding being the main symptom. No patients had a hormonal syndrome. The mean size of the lesions was 21 mm (6 mm to 35 cm). Six (50%) patients had a well-differentiated, benign lesion, 6 (50%) patients had a well-differentiated NET with unknown malignant potential (gangliocytic paragangliomas). Eight (67%) were completely excised during the initial endoscopy with 4 requiring re-excision. Only 2 patients developed recurrence, after 2.5 and 10 years and this necessitated a pancreaticoduodenectomy. Five patients had complications (2 for bleeding and 3 for post-ERCP pancreatitis), with zero deaths. Conclusions: Unlike duodenal carcinoids, ampullary NET are rare. Pancreaticoduodenectomy can be offered to fit patients except for gangliocytic paragangliomas that do not require an aggressive operation. However, for those that refuse surgery or are poor candidates, endoscopic ampullectomy can be an option with acceptable short and long-term outcomes.

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